Iron overload

Beta-thalassemia Market to Witness Upsurge in Growth During the Study Period (2019-2032), Evaluates DelveInsight | Leading Companies - Vertex Pharmaceuticals, CRISPR Therapeutics, Agios Pharmaceuticals, Celgene, Forma Therapeutics

Retrieved on: 
Wednesday, January 17, 2024

LAS VEGAS, Jan. 17, 2024 /PRNewswire/ -- DelveInsight's Beta-thalassemia Market Insights report includes a comprehensive understanding of current treatment practices, beta-thalassemia emerging drugs, market share of individual therapies, and current and forecasted market size from 2019 to 2032, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan].

Key Points: 
  • According to DelveInsight's analysis, the market size for beta-thalassemia across the 7MM is expected to grow with a significant CAGR by 2032.
  • The promising beta-thalassemia therapies in the pipeline include CTX001, EDIT-301, Mitapivat, ACE-536, Panobinostat, Etavopivat tablets, and others.
  • In September 2023, Pharmacosmos has initiated a Phase II trial of SP-420 in patients with transfusion-dependent β-thalassemia.
  • In June 2023, FDA accepted the Biologics License Application (BLAs) of exagamglogene autotemcel (exa-cel) for transfusion-dependent beta thalassemia (TDT).

Beta-thalassemia Market to Witness Upsurge in Growth During the Study Period (2019-2032), Evaluates DelveInsight | Leading Companies - Vertex Pharmaceuticals, CRISPR Therapeutics, Agios Pharmaceuticals, Celgene, Forma Therapeutics

Retrieved on: 
Wednesday, January 17, 2024

LAS VEGAS, Jan. 17, 2024 /PRNewswire/ -- DelveInsight's Beta-thalassemia Market Insights report includes a comprehensive understanding of current treatment practices, beta-thalassemia emerging drugs, market share of individual therapies, and current and forecasted market size from 2019 to 2032, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan].

Key Points: 
  • According to DelveInsight's analysis, the market size for beta-thalassemia across the 7MM is expected to grow with a significant CAGR by 2032.
  • The promising beta-thalassemia therapies in the pipeline include CTX001, EDIT-301, Mitapivat, ACE-536, Panobinostat, Etavopivat tablets, and others.
  • In September 2023, Pharmacosmos has initiated a Phase II trial of SP-420 in patients with transfusion-dependent β-thalassemia.
  • In June 2023, FDA accepted the Biologics License Application (BLAs) of exagamglogene autotemcel (exa-cel) for transfusion-dependent beta thalassemia (TDT).

Bond Biosciences Announces Successful Pre-IND Meeting with the FDA to Advance the Development of BBI-001 for Treatment of Iron Overload Associated with Hereditary Hemochromatosis

Retrieved on: 
Thursday, January 11, 2024

BBI-001 is a novel investigational new drug.

Key Points: 
  • BBI-001 is a novel investigational new drug.
  • It is a non-absorbed, oral therapeutic designed to rapidly form a strong and selective iron chelate in the digestive tract, thus inhibiting iron absorption in patients with symptomatic iron overload due to HH.
  • The structure and properties of BBI-001 have been designed to restrict it to the GI tract to inhibit systemic expose.
  • “FDA was very thoughtful in reviewing the Bond briefing package and replying in depth on the proposed non-clinical and clinical development program options, to facilitate the next Phase of clinical development” observed Curtis L. Scribner, MD, Bond’s Chief Medical Officer.

Keros Therapeutics Presents Clinical Data from its KER-050 Program at the 65th American Society of Hematology Annual Meeting and Exposition

Retrieved on: 
Monday, December 11, 2023

“Additionally, we are encouraged by the preliminary data from the lowest three dose cohorts from our ongoing Phase 2 clinical trial in MF.

Key Points: 
  • “Additionally, we are encouraged by the preliminary data from the lowest three dose cohorts from our ongoing Phase 2 clinical trial in MF.
  • Data for hematological response and markers of hematopoiesis were presented from exploratory analyses of these mITT24 patients.
  • All data presented from this trial is as of the data cut-off date.
  • 13 of those 18 patients (72.2%) achieved TI for at least 24 weeks over the first 48 weeks of treatment.

Alterity Therapeutics Announces Presentation of New Data Demonstrating Novel Mechanisms of ATH434

Retrieved on: 
Thursday, November 16, 2023

The poster entitled, “Potent Antioxidant and Mitochondrial-protectant Effects of ATH434, a Novel Inhibitor of α-Synuclein Aggregation with Moderate Iron-binding Affinity,” presents new data indicating that ATH434 can preserve mitochondrial function after oxidative injury and exert direct anti-oxidant activity independent of its iron binding properties.

Key Points: 
  • The poster entitled, “Potent Antioxidant and Mitochondrial-protectant Effects of ATH434, a Novel Inhibitor of α-Synuclein Aggregation with Moderate Iron-binding Affinity,” presents new data indicating that ATH434 can preserve mitochondrial function after oxidative injury and exert direct anti-oxidant activity independent of its iron binding properties.
  • These features were not observed with another iron binding agent approved for treating iron overload that was also investigated.
  • David Stamler, M.D., Chief Executive Officer of Alterity, commented, “These exciting new data underscore the potential of ATH434 as a treatment for neurodegenerative diseases, including Parkinson’s disease and related disorders.
  • We have long known that ATH434 is able to reduce labile iron which, when elevated, can drive oxidative stress.

Geron Announces IMerge Phase 3 Presentations at ASH Highlighting Significant Durability of Transfusion Independence and Breadth of Effect Across MDS Subgroups with Imetelstat in Lower Risk MDS

Retrieved on: 
Monday, December 11, 2023

The results showed that imetelstat consistently had higher RBC-TI response rates than placebo across these different risk subgroups.

Key Points: 
  • The results showed that imetelstat consistently had higher RBC-TI response rates than placebo across these different risk subgroups.
  • Overall, durable 24-week and 1-year RBC-TI responses were observed with imetelstat in all lower- and higher-risk subgroups.
  • This analysis suggests clinical benefit of imetelstat across different molecularly defined subgroups and independent of the underlying molecular mutation pattern.
  • This analysis indicates that achievement of RBC-TI was associated with improved survival, suggesting that transfusion dependence is a modifiable predictor of clinical outcomes in lower risk MDS.

Inflammasome Therapeutics to Enter Clinic with Treatment for Dry AMD

Retrieved on: 
Monday, November 13, 2023

Key Points: 
  • View the full release here: https://www.businesswire.com/news/home/20231113814506/en/
    inflammasome's sustained release implant contains the first of a new class of inflammasome inhibitor drugs, Kamuvudines, also developed by the company.
  • (Photo: Business Wire)
    In GA, the macula - the part of the retina responsible for central vision - slowly atrophies.
  • They only modestly slow the progression of the disease and increase the risk of developing wet AMD.
  • The start of the trial represents the next step in a decade-long research effort led by Inflammasome Therapeutics’ co-founder, Dr. Jayakrishna Ambati, initiated while he was at the University of Kentucky.

Doctors at Fortis Memorial Research Institute (FMRI), Gurugram give new lease of life to 3 young Iraqi thalassemia patients from same family via rare BMT Procedure

Retrieved on: 
Thursday, October 12, 2023

The family of the 3 Iraqi patients was counseled and explained in detail about all the pros and cons pertaining to stem cell transplant.

Key Points: 
  • The family of the 3 Iraqi patients was counseled and explained in detail about all the pros and cons pertaining to stem cell transplant.
  • After getting informed consent, all three were admitted for a matched sibling donor stem cell transplant, which went off successfully.
  • Speaking about the rare BMT procedure, Dr Vikas Dua said, "The uniqueness about these 3 patients was their younger age.
  • Fortis Gurugram has a dedicated team of Hematologists alongwith the best of medical infrastructure to treat the most complex cases.

Doctors at Fortis Memorial Research Institute (FMRI), Gurugram give new lease of life to 3 young Iraqi thalassemia patients from same family via rare BMT Procedure

Retrieved on: 
Thursday, October 12, 2023

The family of the 3 Iraqi patients was counseled and explained in detail about all the pros and cons pertaining to stem cell transplant.

Key Points: 
  • The family of the 3 Iraqi patients was counseled and explained in detail about all the pros and cons pertaining to stem cell transplant.
  • After getting informed consent, all three were admitted for a matched sibling donor stem cell transplant, which went off successfully.
  • Speaking about the rare BMT procedure, Dr Vikas Dua said, "The uniqueness about these 3 patients was their younger age.
  • Fortis Gurugram has a dedicated team of Hematologists alongwith the best of medical infrastructure to treat the most complex cases.

Bond Biosciences Announces Positive Phase Ia/b Pharmacodynamic and Safety Data for BBI-001, a Novel, Non-Absorbed, Oral Therapy for Iron Overload

Retrieved on: 
Thursday, July 20, 2023

Bond Biosciences (“Bond” or “the Company”) announced positive clinical results today from its 24-person Phase 1a/b, randomized, double-blind, placebo-controlled, single-ascending-dose study that assessed the safety, tolerability, and pharmacodynamic activity of BBI-001, a first-in-class, non-absorbed oral treatment in development for the treatment of iron overload.

Key Points: 
  • Bond Biosciences (“Bond” or “the Company”) announced positive clinical results today from its 24-person Phase 1a/b, randomized, double-blind, placebo-controlled, single-ascending-dose study that assessed the safety, tolerability, and pharmacodynamic activity of BBI-001, a first-in-class, non-absorbed oral treatment in development for the treatment of iron overload.
  • BBI-001 was well-tolerated and exhibited no drug-related adverse events while significantly reducing dietary iron absorption.
  • HH is a genetic disorder characterized by excessive intestinal absorption of iron from food resulting in a pathological increase in total body iron.
  • The current standard of care is to decrease whole body iron stores by periodically removing two units of blood (therapeutic phlebotomy).